BIO CEO 2012 Presenting Company Descriptions

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Below please find descriptions provided by company presenters, categorized by therapeutic category.

Autoimmune/Inflammation  |   Cardiovascular Diseases  |  CNS  |  Dermatology
Drug Discovery
  |  Gastrointestinal  |   Genetic Disorders   |   Hematology  |   Infectious Diseases  |  Metabolic Diseases  |  Oncology  |  Platform  |   Renal  |  Regenerative Medicine  |  Vaccines

 

Autoimmune/Inflammation

Rigel Pharmaceuticals, Inc. Rigel Pharmaceuticals, Inc. is a clinical-stage drug development company that discovers and develops novel, small-molecule drugs for the treatment of inflammatory and autoimmune diseases, as well as muscle disorders. Rigel's pioneering research focuses on intracellular signaling pathways and related targets that are critical to disease mechanisms. Rigel's productivity has resulted in strategic collaborations with large pharmaceutical partners to develop and market its product candidates. Current product develop- ment programs include fostamatinib (R788), an oral syk inhibitor that has started its phase 3 clinical trial program for rheumatoid arthritis (w/AstraZeneca), and R343, an inhaled syk inhibitor that has completed Phase 1 clinical trials for asthma.

Cardiovascular Diseases

Resverlogix Corp. Resverlogix Corp. (TSX:RVX) is a leading biotechnology company developing novel therapies for global medical markets with significant unmet needs. They are developing novel small molecules that enhance ApoA-I production to treat Atherosclerosis, Acute Coronary Syndrome, Alzheimer’s disease, Peripheral Artery Disease, and Autoimmune diseases. Lead drug RVX-208 is currently in two Phase 2b clinical trials led by Cleveland Clinic.

CNS

Allon Therapeutics Inc. Allon Therapeutics Inc. is a clinical-stage biotechnology company focused on bringing to market innovative central nervous system therapies. Allon's lead drug, davunetide, is proceeding in a pivotal Phase 2/3 clinical trial in an orphan indication, progressive supranuclear palsy (PSP), under a Special Protocol Assessment (SPA) with the Food and Drug Administration (FDA). This pivotal trial is based upon statistically significant human efficacy demonstrated in amnestic mild cognitive impairment, cognitive impairment associated with schizophrenia, and positive biomarker data.

ALS Therapy Development Institute Nonprofit biotechnology institute focused on the discovery and development of effective treatments for motor neuron disease (ALS/MND).  Operates a state-of-the-art institute with 30 FTE scientists and collaborates with academic leaders as well as industry.  Currently 6 pharma partnerships.

BioLineRX, Ltd. BioLineRx Ltd. is a publicly-traded biopharmaceutical development company. BioLineRx’s current portfolio consists of five clinical stage candidates: BL-1020 for schizophrenia is in Phase II/III clinical trials; BL-1040 for prevention of pathological cardiac remodeling following a myocardial infarction has completed a Phase I/II study and has been out-licensed to Ikaria Inc. for a total deal value of $282.5 million, in addition to sales royalties; BL-5010 for non-surgical removal of skin lesions has completed a Phase I/II study; BL-1021 for neuropathic pain is in Phase I trials; and BL-7040 for treating Inflammatory Bowel Disease (IBD) has completed Phase I. In addition, BioLineRx has nine products in various pre-clinical development stages for a variety of indications, including central nervous system diseases, oncology, infectious diseases, cardiovascular and autoimmune diseases.

Neurocrine Biosciences, Inc. Neurocrine  Biociences, Inc. is a biopharmaceutical company focused on neurological and endocrine diseases and disorders.  Their product candidates address some of the largest pharmaceutical markets in the world. 

NuPathe Inc. NuPathe Inc. (www.nupathe.com) is an emerging biopharmaceutical company focused on innovative neuroscience solutions for diseases of the central nervous system including neurological and psychiatric disorders. NuPathe's lead product candidate,NP101, is an active, single-use, transdermal sumatriptan patch being developed for the treatment of migraine. In addition to NP101, NuPathe has two additional proprietary product candidates in preclinical development: NP201 for the continuous symptomatic treatment of Parkinson's disease, which the company plans to partner, and NP202 for the long-term treatment of schizophrenia and bipolar disorder.

The Michael J. Fox Foundation for Parkinson's Research As the world’s largest private funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program, and has invested over $270 million in research to date.  www.michaeljfox.org.

Rett Syndrome Research Trust RSRT is the premier organization devoted exclusively to promoting international research on Rett Syndrome and related MECP2 disorders. Their goal is clear: to heal children and adults who will otherwise suffer from this disorder for the rest of their lives.

Titan Pharmaceuticals, Inc.  Titan Pharmaceuticals, Inc is a biopharmaceutical company focused in the development of products for the treatment of central nervous system (CNS) diseases. Titan's first out licensed product, Fanapt® (iloperidone) is an atypical antipsychotic approved in the U.S. in May 2009 for the treatment of schizophrenia in adult patients. Titan's second product, Probuphine, is designed to deliver six months of continuous round-the-clock, long-term therapeutic levels of the drug buprenorphine following a single subcutaneous treatment. Probuphine was developed using ProNeura™, Titan’s continuous drug delivery system that consists of a small, solid rod made from a mixture of ethylene-vinyl acetate (EVA) and a drug substance.

Dermatology

DUSA Pharmaceuticals, Inc. DUSA Pharmaceuticals, Inc. is an integrated dermatology pharmaceutical company focusted primarily on the development & marketing of its Levulan® PDT tehcnology platform and other dermatology products. Levulan® Kerastick® for topical solution plus DUSA’s BLU-U® Blue Light Photodynamic Therapy Illuminator is currently approved for the treatment of minimally to moderately thick actinic keratoses (AKs) of the face or scalp.

Drug Discovery

Cytokinetics, Inc. Cytokinetics is focused on the discovery and development of novel small molecule therapeutics that modulate muscle function.

Lexicon Pharmaceuticals Inc. Lexicon Pharmaceuticals is a biopharmaceutical company focused on discovering and developing breakthrough treatments for human disease.  Using its proprietary gene knockout technology, Lexicon has discovered more than 100 promising drug targets, and created a unique, growing clinical pipeline in the fields of cardiology, gastroenterology, immunology, metabolism, and ophthalmology.

Gastrointestinal

NPS Pharmaceuticals, Inc. NPS Pharmaceuticals is an outsourcing-based development company focused on bringing biopharmaceuticals to patients with rare disorders and few, if any, therapeutic options.  The company is advancing two Phase 3 registration programs, GATTEX® (teduglutide) in adult short bowel syndrome (SBS) and NATPARATM (parathyroid hormone 1-84 [rDNA origin] injection) in hypoparathyroidism.  NPS’ earlier stage pipeline includes two calcilytic compounds, NPSP790 and NPSP795, with potential application in rare disorders involving increased calcium receptor activity, such as autosomal dominant hypocalcemia with hypercalciuria (ADHH).  NPS complements its proprietary programs with a royalty-based portfolio of products and product candidates.

Tranzyme, Inc. Tranzyme Pharma is a late-stage biopharmaceutical company focused on discovering, developing and commercializing novel, mechanism-based therapeutics for the treatment of upper gastrointestinal (GI) motility disorders. Tranzyme is developing an intravenous drug, ulimorelin, for patients in acute (hospital-based) settings, as well as an oral drug (TZP-102) for chronic conditions. Ulimorelin is currently in Phase 3 clinical trials and TZP-102 is entering Phase 2b. Together these product candidates target a significant underserved market.

Genetic Disorders

BioMarin Pharmaceutical Inc. BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates.

Hematology

Cerus Corporation Cerus is a developing and commercializing novel, proprietary products and technologies that are intended to make the blood supply safer. We are commercializing the INTERCEPT Blood System for platelets and plasma in Europe, Russia, the Middle East and selected countries in other regions around the world. The INTERCEPT Blood System for platelets and plasma is not yet approved for sale in the United States. In 2008, Cerus initiated a Phase I clinical trial for their INTERCEPT Blood System for red blood cells in the United States. The INTERCEPT Blood System, which is based on their proprietary Helinx technology for controlling biological replication, is designed to enhance the safety of donated blood components by inactivating viruses, bacteria, parasites and other pathogens, as well as potentially harmful white blood cells.

Infectious Diseases

Idenix Pharmaceuticals, Inc. Idenix Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts, is a biopharmaceutical Company engaged in the discovery and development of drugs for the treatment of human viral diseases. Idenix's current focus is on the treatment of patients with chronic hepatitis C infection.

PolyMedix Inc. PolyMedix is a clinical stage biotechnology company developing first-in-class small-molecule drugs for the treatment of acute care conditions. Based on their proprietary drug discovery platform, the company has created a pipeline of innovative infectious disease and cardiovascular product candidates.

Metabolic Diseases

GENFIT A biopharmaceutical company in the diabetes area, GENFIT is focused on the diagnosis and treatment of diabetes related complications, including liver and cardiovascular diseases. GFT505, GENFIT's most advanced drug candidate, currently in phase II, is well positioned to become a first-in-class therapy for NAFLD/ NASH. GENFIT seeks Investors to fund the late-stage clinical development of GFT505 and support its optimal progress & value.

Theratechnologies Inc. Theratechnologies (TSX: TH)(NASDAQ: THER) is a specialty pharmaceutical company that discovers and develops innovative therapeutic peptide products. Its lead product, EGRIFTA® (tesamorelin for injection), is an analogue of the human growth hormone releasing factor.

Oncology

Azanta A/S Azanta is a Danish based development and specialty Pharma Company with sales organization in Scandinavia, UK and Canada. Azanta develops its own phase III projects in Head and Neck cancer for font line treatment in combination with radiation. Azanta has 3 additional internal development projects with special license sales potential within the next 1-2 years.

Celsion Corporation Celsion Corporation (NASD: CLSN) is a clinical stage (Phase III) biopharmaceutical company singularly focused on developing its unique heat activated liposomal technology to create more targeted, efficacious versions of marketed cancer therapeutics. The company aims to develop ThermoDox®, its proprietary thermally sensitive liposomal encapsulation of doxorubicin to NDA for 4 unmet need indications: HCC (Hepatocellular Carcinoma) or primary liver cancer; RCW (Recurrent Chest Wall) breast cancer;  CRLM (Colorectal Liver Metastases);  and, Bone Mets (Prostate Bone Metastases). On the business and partnering side, the company will 1) Identify opportunities to expand the range of therapeutic indications and drug candidates through investments in it’s core technology platform; 2) Establish Ex-US commercialization alliances that ensure broad global distribution through either regional (i.e. Asia Pacific or Pan European) or country-specific (i.e. Japan with Yakult Honsha) licensing and distribution agreements; and 3)  Establish US sales and marketing capability through either the acquisition of small to mid-size oncology drugs, product lines, or businesses; or build-out of a full-service oncology enterprise exclusive targeted at the US oncology and interventional markets.

Cyclacel Pharmaceuticals, Inc. Cyclacel is  a biopharmaceutical company developing oral therapies that target various phases of cell cycle control for the treatment of cancer and other serious diseases.

Delcath Systems, Inc. Delcath Systems, Inc. is a development stage specialty pharmaceutical and medical device company focused on oncology. Their proprietary system for chemosaturation is designed to administer high dose chemotherapy and other therapeutic agents to diseased organs or regions of the body, while controlling the systemic exposure of those agents. Delcath's initial focus is on the treatment of primary and metastatic liver cancers. They concluded a Phase III metastatic melanoma study, and recently completed a multi-arm Phase II trial to treat other liver cancers. Delcath received CE Mark approval for the Hepatic CHEMOSAT delivery system in April 2011.

Infinity Pharmaceuticals Infinity, www.infi.com, is an innovative drug discovery and development company dedicated to discovering, developing and delivering to patients best-in-class medicines for difficult-to-treat diseases.

MannKind Corporation MannKind Corporation (Nasdaq: MNKD) focuses on the discovery, development and commercialization of therapeutic products for patients with diseases such as diabetes and cancer. Its diabetes pipeline includes AFREZZA®, an ultra rapid-acting prandial insulin and MKC253, an inhaled formulation of human GLP-1.  MannKind also has two cancer immunotherapeutic products in clinical development. 

MethylGene MethylGene is a small molecule drug development company with two programs moving into mid stage studies, MGCD265 for oncology and MGCD290 for the treatment of fungal infections. The company has a portfolio of more than 12,000 proprietary, rationally-designed, small molecule inhibitors of important targets for oncology, infectious diseases and other major indications. MethylGene completed a C$34.5 million financing in April of 2011 with thought-leading life sciences investors and recently reported C$33 million in cash (Sept. 30, 2011). MethylGene is publicly-traded on the Toronto Stock Exchange under the symbol MYG.

Onconova Therapeutics, Inc. Onconova Therapeutics is a Phase III stage development company focused on novel cancer and radiation protection drugs. NDA filing of rigosertib (Estybon, ON 01910.Na) planned for 2013. Rigosertib addresses hard to treat cancers including MDS, pancreatic and ovarian cancers. They are a private company located in the Princeton, NJ area with a chemistry based platform and a pipeline of small molecule drugs aimed at novel targets.

OncoSec Medical Inc. OncoSec Medical Inc. is an advance stage biomedical company developing therapeutics to selectively destroy cancer tumors. OMI’s core technology is a novel delivery system using a form of cell membrane stimulation known as electroporation. Using this system to deliver a proven Chemotherapy or Immunotherapy, these therapies can effectively treat cancerous tumors while reducing or eliminating detrimental treatment outcomes associated with other “standard of care” therapies.

Peregrine Pharmaceuticals, Inc. Peregrine is a clinical-stage biopharmaceutical company developing first-in-class monoclonal antibodies for the treatment of cancer and viral infections.

Provectus Pharmaceuticals Inc. Provectus Pharmaceuticals specializes in developing oncology and dermatology therapies. Its novel oncology drug PV-10 is designed to selectively target and destroy cancer cells without harming surrounding healthy tissue, significantly reducing potential for systemic side effects. Its dermatological drug PH-10 also targets abnormal or diseased cells, with the current focus on psoriasis and atopic dermatitis.

Selvita S.A. Selvita is a Polish biotechnology company with a focus on the discovery and development of innovative, breakthrough medicines to treat CNS, oncology and autoimmune disorders. Selvita presents a hybrid model of activity, joining innovative drug discovery  with provision of custom non-clinical research services. The company was established in 2007 and currently employs 135 people including 50 PhDs.

Sunesis Pharmaceuticals, Inc. Sunesis is a biopharmaceutical company focused on the development and commercialization of new oncology therapeutics for the treatment of solid and hematologic cancers. Sunesis’ lead product candidate, vosaroxin, a first-in-class anticancer quinolone derivative, is currently under evaluation in the Phase 3 VALOR trial, a randomized, double-blind, placebo-controlled, pivotal trial in patients with first relapsed or refractory acute myeloid leukemia (AML). Sunesis is also developing kinase inhibitors in oncology through a collaboration with Millennium Pharmaceuticals, Inc., under which they recently initiated a Phase 1 clinical trial of MLN2480, an oral investigative drug selective for pan-Raf kinase inhibition.

Telik, Inc. Telik, Inc. is a biopharmaceutical company dedicated to discovering, developing and commercializing novel small molecule drugs to treat cancer. The lead candidate is TELINTRA® (TLK199) which is currently in Phase 2 clinical development for myelodysplastic syndrome, and severe chronic idiopathic neutropenia. TELCYTA® (TLK286 ), is a cancer cell-activated chemotherapeutic that has shown clinical activity in Phase 2 and Phase 3 clinical trials. Product candidates in our pipeline were discovered using our proprietary technology, TRAP®, which enables the rapid and efficient discovery of small molecule drug candidates. Telik was founded in 1988 and is headquartered in Palo Alto, CA.

Verastem, Inc. Verastem, Inc. is a private biopharmaceutical company focused on discovering and developing novel drugs that selectively target cancer stem cells. Cancer stem cells are an underlying cause of tumor recurrence and metastasis. Verastem is translating discoveries in cancer stem cell research into new medicines for the treatment of major cancers such as breast cancer.

ZIOPHARM Oncology, Inc.  ZIOPHARM Oncology, Inc. is a biopharmaceutical company that is seeking to develop and commercialize a diverse portfolio of cancer drugs. Their focus is on the development of proprietary small molecule drug candidates that work on validated targets as well as the development of novel DNA-based therapeutics through their exclusive channel partnership in oncology with Intrexon Corporation. The goal is to develop intravenous ("IV") and/or oral forms of administration. The company believes this strategy will result in lower-risk and lower-cost development programs with product candidates having a low cost of manufacturing to address changing reimbursement requirements around the world.

Oncology & Autoimmune Disease

Compugen Compugen is a leading therapeutic product candidate discovery company, currently focused on biologics-based therapy to address important unmet needs in the fields of immunology and oncology. Compugen’s discovery efforts are based on systematic and continuously improving in-silico (by computer) product candidate prediction and selection followed by experimental validation, with selected product candidates being advanced in its Pipeline Program to the pre-IND stage. The Company’s business model primarily involves collaborations covering the further development and commercialization of Compugen-discovered product candidates and various forms of “discovery on demand” arrangements, in both cases providing Compugen with potential milestone payments and royalties on product sales or other forms of revenue sharing.

Oncology & CNS

e-Therapeutics plc e-Therapeutics (AIM: ETX) pioneered and exploits network pharmacology as a drug discovery platform. The Company is advancing four drugs in the clinic, in oncology, infection and psychiatry, and plans to partner these after mid-phase trials. New discovery projects at Oxford are focused in oncology and degenerative diseases.

Oncology & Fibrosis

Galectin Therapeutics Galectin Therapeutics (OTC: GALT) is developing promising carbohydrate-based therapies for fibrotic liver disease and cancer based on the company's unique understanding of galectin proteins, key mediators of biologic function. We are leveraging extensive scientific and development expertise as well as established relationships with external sources to achieve cost effective and efficient development. We are pursuing a clear development pathway to clinical enhancement and commercialization for our lead compounds in liver fibrosis and cancer.

Oncology & Hematology

ADVENTRX Pharmaceuticals, Inc. ADVENTRX Pharmaceuticals is a specialty pharmaceutical company focused on developing proprietary product candidates. The company’s current lead product candidates are ANX-188, a novel, purified, rheologic and antithrombotic compound initially being developed as a first-in-class treatment for pediatric patients with sickle cell disease in acute crisis, and ANX-514, a novel, detergent-free formulation of the chemotherapy drug docetaxel.

Oncology & Infectious Disease

Agenus Inc. Agenus is a biotechnology company working to develop treatments for cancers and infectious diseases. The company is focused on immunotherapeutic products based on strong platform technologies with multiple product candidates advancing through the clinic, including several product candidates that have advanced into late-stage clinical trials through corporate partners. Between Agenus and its partners, 18 programs are in clinical development. For more information, please visit www.agenusbio.com.

Oncology & Women's Health

BioSante Pharmaceuticals, Inc. BioSante is a specialty pharmaceutical company focused on developing products for female sexual health and oncology. BioSante's lead products include LibiGel® (transdermal testosterone gel) for the treatment of female sexual dysfunction (FSD), specifically hypoactive sexual desire disorder (HSDD), which is in Phase III clinical development according to a U.S. Food and Drug Administration (FDA) Special Protocol Assessment (SPA). BioSante's first FDA-approved product is Elestrin™ (estradiol gel) indicated for the treatment of hot flashes associated with menopause, marketed in the U.S. by Azur Pharma, BioSante's licensee. BioSante also is developing a portfolio of cancer vaccines, four of which have been granted Orphan Drug designation, and are currently in several Phase II clinical trials. Other BioSante products are Bio-T-Gel™, a testosterone gel for male hypogonadism, for which an NDA is pending, licensed to Teva Pharmaceuticals, and an oral contraceptive in Phase II clinical development. Additional information is available online at: www.biosantepharma.com.

Platform

KineMed KineMed’s decision relevant information guides pharma R&D. Its Translational Biomarker Platform pin-points the function of thousands of proteins, all in a drop of blood,  capturing causal process  driving disease progression, and predicting clinical outcomes. KineMed successfully collaborates with leading investigators to enable better medicines and power personalized medicine.

Renal

Affymax Affymax is a biopharmaceutical company committed to developing novel drugs to improve the treatment of serious and often life-threatening conditions. The company’s first product candidate, peginesatide (formerly known as Hematide™), has been investigated in Phase 3 trials for the potential treatment of anemia associated with chronic kidney disease (CKD). A New Drug Application (NDA) for peginesatide for the treatment of anemia associated with CKD in adult patients on dialysis is currently being reviewed by the U.S. Food and Drug Administration (FDA).

Regenerative Medicine

Athersys Inc. Athersys, Inc. (NASDAQ: ATHX) is recognized as a leader in the field of regenerative medicine.  The company is developing a patented & proprietary stem cell therapy called MultiStem, which offers a distinctive combination of manufacturing scalability, multi-modal therapeutic potential, and ease of administration.  Athersys has 4 clinical stage programs in areas including cardiovascular disease, neurological conditions, inflammatory & immune disease, and transplant support, and is partnered with Pfizer as well as leading academic and clinical institutions.

NeoStem, Inc. NeoStem is a leader in the development and manufacture of cell therapies. NeoStem has a strategic combination of revenues, including that which is derived from the contract manufacturing services performed by Progenitor Cell Therapy, LLC, a NeoStem company. That manufacturing base is one of the few cGMP facilities available for contracting in the burgeoning cell therapy industry. Amorcyte, Inc., also a NeoStem company, is developing a cell therapy for the treatment of cardiovascular disease and its lead compound is poised to commence enrollment in a Phase 2 trial for the preservation of heart function after a heart attack.

Vaccines

Immunovaccine Inc. Immunovaccine Inc., (TSX-V: IMV) develops vaccines formulated in its vaccine adjuvanting platform for cancer therapy and infectious disease. The Company’s DepoVax™ platform is a patented lipid delivery system that presents antigens plus adjuvant to the immune system for a prolonged period and has the potential to enhance immune responses. Immunovaccine has taken its platform technology and proprietary cancer vaccine into Phase I human trials and has demonstrated its safety and immunogenicity potential. The Company is also capitalizing on the broad potential of its delivery platform by creating new DepoVax-based vaccines through multiple development collaborations. In addition to the Company’s human health vaccine strategy, it continues to capture value from animal health vaccine applications. Pfizer Animal Health has licensed the Company’s delivery technology platform to develop vaccines for livestock. www.imvaccine.com

Medicago Inc. Medicago (TSX: MDG) is a clinical-stage biotechnology company focused on developing highly effective and competitive vaccines based on proprietary manufacturing technologies and Virus-Like Particles (VLPs). Medicago’s VLP manufacturing technology is based on a transient expression system which produces recombinant vaccine antigens in non-transgenic plants. It can deliver a vaccine for testing in less than a month after the identification and reception of genetic sequences from a pandemic strain. Medicago recently reported positive results from its Phase II clinical trial with its avian flu H5 pandemic vaccine candidate, and also reported positive results from a U.S. Phase I human clinical trial with its seasonal influenza vaccine candidate.

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For the full list of presenting companies at the 14th annual BIO CEO & Investor Conference, including websites and stock information, please visit our 2012 Presenting Companies page.

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